| |
|
BENEFIX Novel Hemophilia B Therapy Receives FDA License
CAMBRIDGE, Mass., Feb. 12, 1997 -- Genetics Institute, Inc., a wholly-owned subsidiary of American Home Products Corporation (NYSE: AHP), announced today that the U.S. Food and Drug Administration (FDA) has licensed BENEFIX(TM), Coagulation Factor IX (Recombinant), a clotting factor used to treat hemophilia B. Made through biotechnology, plasma-free, albumin-free BENEFIX is a breakthrough product. For the first time, patients will have access to a product that is inherently free from the risk of transmission of human blood borne pathogens such as HIV, hepatitis viruses and parvovirus. This has not been previously possible with products derived from human blood. BENEFIX is manufactured and distributed by Genetics Institute in the United States and distributed by Baxter Healthcare Corporation in Europe.
"Having a recombinant Factor IX product available represents a significant safety advance for patients with hemophilia B," said Stephen E. Bajardi, Executive Director of the National Hemophilia Foundation, a non-profit voluntary health agency representing the interests of people with hemophilia.
The Need for a Recombinant Factor IX Treatment
Hemophilia B is an inherited disorder that almost exclusively affects males and is caused by a deficiency or defect in Factor IX, one of a number of proteins involved in blood clotting. Factor IX deficiency results in frequent hemorrhages that can be crippling and, at times, life-threatening. The disorder affects about 7,000 people in North America, Europe and Japan (approximately 3,300 patients in the U.S.). BENEFIX has Orphan Drug designation in the United States.
Factor IX products available until now have all been made from human blood. While earlier Factor IX products have been specifically treated to reduce the possible transmission of human pathogenic viruses, the risk of viral infectivity from these products has not been totally eliminated. Since BENEFIX Coagulation Factor IX (Recombinant) is made through recombinant technology, without the addition of blood or plasma products, it eliminates the risk of blood-borne viral contamination. Further, the supply of BENEFIX does not depend on blood donors. Genetics Institute expects to be able to manufacture product at levels sufficient to meet worldwide demand.
Summary of Clinical Studies
"We found BENEFIX to be effective for the treatment of hemophilia B, including spontaneous bleeds and surgery," said Dr. Gilbert White, Professor of Medicine and Pharmacology at the University of North Carolina and one of the investigators in the clinical studies with BENEFIX. "The licensure of BENEFIX is an important milestone in the treatment of hemophilia B," he added.
Clinical trials demonstrated the efficacy of BENEFIX. In the trials, it was found that some patients may require higher doses of BENEFIX compared to plasma-derived products. The trials also showed that BENEFIX had a safety profile comparable to other Factor IX products.
Due to the rarity of hemophilia B, clinical studies of BENEFIX have involved only a small number of patients. Few data are available regarding the rare, serious side effects that occur from treatment with factor IX products. These side effects include antibody responses that neutralize the effect of factor IX, or thrombosis due to excessive blood clotting. Genetics Institute will follow for two years 36 patients receiving BENEFIX who had previously received plasma-derived products. Specifically, the Company will monitor for major thrombotic events, inhibitor formation, and other adverse events. In addition, since information regarding use of BENEFIX in previously untreated patients currently is limited, Genetics Institute is continuing a clinical trial involving up to 50 previously untreated patients for up to five years to evaluate this patient population for safety, efficacy, and inhibitor formation.
Genetics Institute's History in Providing Hemophilia Therapeutics
"Genetics Institute is proud to use its leading-edge science in developing BENEFIX to assist the hemophilia community," said Patrick Gage, Ph.D., President of Genetics Institute. "We began our commitment to develop breakthrough products for this community with our work in the early 1980s on the first licensed recombinant Factor VIII product, and we continue that commitment with the launch of the first and only recombinant Factor IX product," he said.
Genetics Institute is also the originator and bulk-drug supplier of a recombinant Factor VIII product. Recombinate, Antihemophilic Factor (Recombinant) is distributed in North America, Europe and Japan by the Company's licensee, Baxter Healthcare Corporation, Hyland Division.
Genetics Institute's First Proprietary Product
BENEFIX is the first product to be marketed and sold directly by the Company. A New Drug Submission for BENEFIX is in review by the Canadian Health Protection Branch and a Marketing Authorization Application is in review by the European Medical Evaluation Agency.
Genetics Institute, Inc., a wholly-owned subsidiary of American Home Products Corporation (NYSE: AHP), is a leading biopharmaceutical firm engaged in the discovery, development, and commercialization of human pharmaceuticals through recombinant DNA and other technologies. AHP is one of the world's largest research-based pharmaceutical and health care products companies, and is a leading developer, manufacturer and marketer of prescription drugs and over-the-counter medications. It is also a leader in vaccines, biotechnology, agricultural products, animal health care and medical devices.
The statements in this release that are not historical facts are forward-looking statements that involve risks and uncertainties, including, without limitation, risks associated with the inherent uncertainty of pharmaceutical research, product development and commercialization, the impact of competitive products, patent, product liability and third party reimbursement risks associated with the pharmaceutical industry, and the other risks and uncertainties detailed from time to time in AHP's periodic reports, including quarterly reports on Form 10-Q and the annual report on Form 10-K, filed with the Securities and Exchange Commission. Actual results may differ from the forward-looking statements.
|
